Romiplostim

Romiplostim (rINN, USAN) is a fusion protein analog of thrombopoietin, a hormone that regulates platelet production.

Safety Concerns vs. Marketing

The drug was developed by Amgen and is marketed under the trade name Nplate through a restricted usage program called NEXUS. During development and clinical trials the drug was called AMG531.

Romiplostim is indicated as a potential treatment for chronic idiopathic (immune) thrombocytopenic purpura (ITP). Romiplostim was designated an orphan drug by the U.S. Food and Drug Administration (FDA) in 2003, as the chronic ITP population in the USA is under 200,000 (the chronic adult ITP population in the USA is thought to be around 60,000, with women outnumbering men by a factor of two). The wholesale cost of romiplostim if administered weekly is currently estimated at US $55,250 per year.

On August 22, 2008, the FDA approved romiplostim as a long-term treatment for chronic ITP in adults who have not responded to other treatments, such as corticosteroids, intravenous immunoglobulin, Rho(D) immune globulin or splenectomy.